The therapies target specific biological pathways known to
drive chronic lymphocytic leukemia, a typically slow-moving
cancer of the blood and bone marrow that usually strikes older
people, some of whom can’t tolerate the toxic chemotherapies
that are part of current treatment regimens.

Data presented on several of the medicines yesterday at the
American Society of Hematology meeting in New Orleans suggest
combination regimens may arise that remove the need for
chemotherapy and its related toxic side effects while driving
better results, said Richard Furman, director of the CLL
Research Center at Weill Cornell Medical College in New York.

The new drugs “herald a dawn of a new age for CLL
patients,” Furman told reporters yesterday in New Orleans.
“This really is, I think, a great opportunity for us to do what
I consider to be the most important thing for our patients:
eliminate chemotherapy from our treatment paradigm.”

The progress being made in CLL is indicative of the way
cancer may increasingly be treated, with combination regimens of
targeted therapies aimed at different biological pathways.
Potential new drugs for CLL include ibrutinib, from
Pharmacyclics and Johnson & Johnson (JNJ); Gilead’s idelalisib;
Infinity Pharmaceuticals Inc. (INFI)’s IPI-145; AbbVie Inc. (ABBV)’s ABT-199;
and Roche’s Gazyva, approved last month.

Chemotherapy Effects

Chemotherapy’s short-term side effects can include
suppression of bone marrow and immune response, fatigue, hair
loss, nausea and vomiting, according to the National Cancer
Institute. It also has the potential to cause longer-term
problems, like additional cancers, said Furman, a lead
investigator in Gilead’s late-stage trial of idelalisib.

“The bar has been set much higher now in terms of where we
should be thinking and what the goals of therapy are,” Furman
said in an interview yesterday. “No longer just getting your
patient into remission but getting your patient into remission
safely. That’s a very nice place to be.”

Idelalisib, from Foster City, California-based Gilead,
performed so much better when given in combination with a Roche
drug, Rituxan, than Rituxan alone that the late-stage trial was
stopped early. The research was presented at the New Orleans
meeting. Analysts expect the drug to draw $1.2 billion in 2018
revenue. Gilead shares gained 1.6 percent to $75.19 at 4 p.m. in
New York trading. Infinity Pharmaceuticals, based in Cambridge,
Massachusetts, rose 2.6 percent to $15.34.

Gazyva Findings

Basel, Switzerland-based Roche’s Gazyva, in a study also
presented in New Orleans, helped patients live almost a year
longer without their disease progressing than Rituxan when both
medicines were given along with the chemotherapy chlorambucil.
Analysts expect the drug will draw 1.4 billion Swiss francs
($1.57 billion) in annual revenue in 2018, according to
estimates compiled by Bloomberg.

IPI-145, an earlier-stage drug from Cambridge,
Massachusetts-based Infinity, showed promise in CLL patients who
had already tried other drugs, according to updated study
results reported yesterday. Almost half of the 27 patients in an
early-stage study responded to the drug, and six of eight
patients who had been on the medicine for at least 12 months
still hadn’t had their cancer progress.

The highest expectation among new CLL drugs is for
ibrutinib, from Pharmacyclics and J&J, said Michael Yee, an
analyst with RBC Capital Markets. The medicine is expected to
draw $4.7 billion in 2019 revenue for Sunnyvale, California-based Pharmacyclics.

Ibrutinib Response

Ibrutinib was approved by the U.S. Food and Drug
Administration last month for mantle cell lymphoma, with a
decision expected for CLL by the end of February. A study
presented at last year’s hematology meeting and published in
June in the New England Journal of Medicine showed that 71
percent of patients responded to the drug. Further data are
being presented at this year’s meeting.

“These agents are both game changers,” Furman said of
ibrutinib and idelalisib. “People who should have died five
years ago are alive and well and in complete remission. It’s a
huge paradigm shift.”

Researchers in New Orleans this year also presented data on
a technology that reprograms human immune cells in the
laboratory to attack leukemia, showing 15 of 32 patients with
CLL experienced a reduction of their cancers and seven achieved
remission. In patients with acute lymphoblastic leukemia, 19 of
22 children experienced complete remission, as did all five
adults tested.

The technology, led by Carl June at the University of
Pennsylvania, has been licensed to Swiss drugmaker Novartis AG
to open international trials.

“This is the year of CLL,” Sandra Horning, head of
oncology at Roche’s Genentech unit, said in an interview
yesterday. “There’s never been a better time for CLL
patients.”